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Journal Articles

High-Risk Population Screening for Gaucher Disease: Final Results and Key Learnings from an Italian Multicenter Observational Study

Irene Motta, MD, Christian Benedetto, Marina Stroppiano, Elena Cassinerio, MD, Dario Consonni, MD, Lorenza Borin, MD, Paola Ranalli, MD, Wilma Barcellini, Maria Domenica Cappellini, MD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-141527

Published: 2020

Journal Articles

Diagnostic Work-up of Pulmonary Hypertension in Non Transfusion Dependent Thalassemia Patients: Pathophysiologichal Mechanisms and Clinical Implications

Marta Mancarella, MD, Irene Motta, MD, Irene Rota, MD, Margherita Migone De Amicis, MD, Alessia Marcon, MD, Marta Ferraresi, MD, Marianna Giuditta, MD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Marco Vicenzi, MD

Blood (2020) 136 (Supplement 1): 21–22.

https://doi.org/10.1182/blood-2020-140819

Published: 2020

Journal Articles

COVID 19 and Hemoglobinopathies: Update of the Italian Experience

Irene Motta, MD, Valeria Maria Pinto, MD, Filomena Longo, MD, Federico Bonetti, MD, Elisa De Michele, MD, Alberto Piperno, MD, Lorella Pitrolo, MD, Micol Quaresima, MD, Michela Ribersani, MD, Margherita Migone De Amicis, MD, Manuela Balocco, MD, Barbara Gianesin, PhD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Lucia De Franceschi, MD, Antonio Piga, MD, Gian Luca Forni, MD

Blood (2020) 136 (Supplement 1): 17–18.

https://doi.org/10.1182/blood-2020-141610

Published: 2020

Journal Articles

Fyn Specifically Regulates the Activity of Red Cell Glucose-6-Phosphate-Dehydrogenase

Blood (2019) 134 (Supplement_1): 3527.

https://doi.org/10.1182/blood-2019-125709

Published: 2019

Journal Articles

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

Paola Delbini, PhD, Viola Ghiandai, Maria Domenica Cappellini, MD, Lorena Duca, Isabella Nava, Elena Cassinerio, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3592.

https://doi.org/10.1182/blood-2019-130032

Published: 2019

Journal Articles

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

Natalia Scaramellini, MD, Carola Arighi, MD, Alessia Marcon, MD, Dario Consonni, MD, Elena Cassinerio, MD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Irene Motta, MD

Blood (2019) 134 (Supplement_1): 3542.

https://doi.org/10.1182/blood-2019-130237

Published: 2019

Journal Articles

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

Blood (2019) 134 (Supplement_1): 3545.

https://doi.org/10.1182/blood-2019-122685

Published: 2019

Journal Articles

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Blood (2019) 134 (Supplement_1): 2244.

https://doi.org/10.1182/blood-2019-127017

Published: 2019

Journal Articles

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

John Porter, Maria Domenica Cappellini, MD, Thomas Coates, MD, Olivier Hermine, Vip Viprakasit, MD DPhil, Ersi Voskaridou, Hong Keng Liew, Silverio Perrotta, Abderrahim Khelif, Antonis Kattamis, Abderrahmane Laadem, Jeevan K. Shetty, Wen-Ling Kuo, Dimana Miteva, Tatiana Zinger, Peter G. Linde, Daniel Sinsimer, Ali Taher

Blood (2019) 134 (Supplement_1): 2245.

https://doi.org/10.1182/blood-2019-122561

Published: 2019

Journal Articles

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Donatella Baronciani, MD, Maddalena Casale, MD, Lucia De Franceschi, MD, Giovanna Graziadei, MD, Filomena Longo, MD, Raffaella Origa, MD, Valeria Maria Pinto, MD, Paolo Rigano, MD, Monia Marchetti, MD, Antonia Gigante, Emanuele Angelucci, MD, Maria Domenica Cappellini, MD, Achille Iolascon, MD PhD, Antonio Piga, Gian Luca Forni

Blood (2019) 134 (Supplement_1): 972.

https://doi.org/10.1182/blood-2019-128320

Published: 2019

Journal Articles

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

Christophe Lechauve, PhD, Julia Keith, Eugene Khandros, MD PhD, Stephanie Fowler, Kalin Mayberry, Abdullah Freiwan, Christopher Thom, MD PhD, Jingjing Zhang, Heather Tillman, DVM, PhD, Mondira Kundu, MD PhD, Irene Motta, MD, Maria Domenica Cappellini, MD, Mitchell J. Weiss, MD PhD

Blood (2018) 132 (Supplement 1): 411.

https://doi.org/10.1182/blood-2018-99-113901

Published: 2018

Journal Articles

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Blood (2018) 132 (Supplement 1): 3633.

https://doi.org/10.1182/blood-2018-99-111770

Published: 2018

Journal Articles

Phase 2 Clinical Trial Results for Bitopertin, an Oral Glycine Transporter 1 Inhibitor, in Patients with Non-Transfusion Dependent Beta-Thalassemia

Ali Taher, MD PhD, Vip Viprakasit, MD DPhil, Maria Domenica Cappellini, MD, Dominik Kraus, PhD, Patrick Cech, PhD, Volz Dietmar, PhD, Erica Winter, PharmD, Norman Alan Mazer, MD PhD, Stephane Nave, MD, Juergen Dukart, PhD, Franziska Schaedeli Stark, PhD, Annette Koerner, PhD, Omar Khwaja, MD PhD, Ricardo Hermosilla, DVM, PhD

Blood (2018) 132 (Supplement 1): 3635.

https://doi.org/10.1182/blood-2018-99-114776

Published: 2018

Journal Articles

The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

Blood (2018) 132 (Supplement 1): 163.

https://doi.org/10.1182/blood-2018-163

Published: 2018

Journal Articles

Physical Activities in Patients with β-Thalassemia As Measured By Fitbit in Association with Patient-Reported Outcomes

Antonis Kattamis, MD, Pranee Sutcharitchan, MD, Ali Taher, MDPhDFRCP, Maria Domenica Cappellini, MD, Dalia Mahmoud, Joseph Pariseau, PharmD, Abderrahmane Laadem, MD, Anzalee Khan, PhD, X. Henry Hu, MD PhD, Vip Viprakasit, MD PhD

Blood (2017) 130 (Supplement 1): 4731.

https://doi.org/10.1182/blood.V130.Suppl_1.4731.4731

Published: 2017

Journal Articles

Quality of Life in Patients with β-Thalassemia: Transfusion Dependent Versus Non-Transfusion Dependent

Maria Domenica Cappellini, MD, Antonis Kattamis, MD, Vip Viprakasit, MD PhD, Pranee Sutcharitchan, MD, Dalia Mahmoud, Joseph Pariseau, PharmD, Abderrahmane Laadem, MD, Anzalee Khan, PhD, X. Henry Hu, MD PhD, Ali Taher, MDPhDFRCP

Blood (2017) 130 (Supplement 1): 751.

https://doi.org/10.1182/blood.V130.Suppl_1.751.751

Published: 2017

Journal Articles

Exploring the Clinical Utility of Renal Safety Biomarkers during Iron Chelation Therapy in Patients with β-Thalassemia and Other Anemias

Maria Domenica Cappellini, MD, Erhard Quebe-Fehling, PhD, Celine Pallaud, Frank Dieterle, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD

Blood (2017) 130 (Supplement 1): 4762.

https://doi.org/10.1182/blood.V130.Suppl_1.4762.4762

Published: 2017

Journal Articles

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Blood (2017) 130 (Supplement 1): 2211.

https://doi.org/10.1182/blood.V130.Suppl_1.2211.2211

Published: 2017

Journal Articles

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Blood (2017) 130 (Supplement 1): 355.

https://doi.org/10.1182/blood.V130.Suppl_1.355.355

Published: 2017

Journal Articles

Digital PCR Assay to Assess the Alternative Splicing and Absolute Ferrochelatase Expression

Valentina Brancaleoni, MSc, Francesca Granata, MSc, Silvia Fustinoni, PhD, Giovanna Graziadei, MD, Maria Domenica Cappellini, MD, Elena Di Pierro, PhD

Blood (2017) 130 (Supplement 1): 3474.

https://doi.org/10.1182/blood.V130.Suppl_1.3474.3474

Published: 2017

NARROW

High-Risk Population Screening for Gaucher Disease: Final Results and Key Learnings from an Italian Multicenter Observational Study

Diagnostic Work-up of Pulmonary Hypertension in Non Transfusion Dependent Thalassemia Patients: Pathophysiologichal Mechanisms and Clinical Implications

COVID 19 and Hemoglobinopathies: Update of the Italian Experience

Fyn Specifically Regulates the Activity of Red Cell Glucose-6-Phosphate-Dehydrogenase

Evaluation of in Vitro Macrophage Characterization in Gaucher Type 1 (GD1) Patients

Iron Chelation and Ferritin below 500 Mcg/L in Transfusion Dependent Thalassemia: Beyond the Limits of Clinical Trials

Evaluating Luspatercept Responders in the Phase 3, Randomized, Double-Blind, Placebo-Controlled BELIEVE Trial of Luspatercept in Adult Beta-Thalassemia Patients (Pts) Who Require Regular Red Blood Cell (RBC) Transfusions

2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies

Effects of Luspatercept on Iron Overload and Impact on Responders to Luspatercept: Results from the BELIEVE Trial

Selecting ß-Thalassemia Patients for Gene Therapy: A Decision-Making Algorithm

Activation of ULK1 Kinase Mediates Clearance of Free Alpha-Globin in Human Beta-Thalassemic Erythroblasts

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Phase 2 Clinical Trial Results for Bitopertin, an Oral Glycine Transporter 1 Inhibitor, in Patients with Non-Transfusion Dependent Beta-Thalassemia

The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions

Physical Activities in Patients with β-Thalassemia As Measured By Fitbit in Association with Patient-Reported Outcomes

Quality of Life in Patients with β-Thalassemia: Transfusion Dependent Versus Non-Transfusion Dependent

Exploring the Clinical Utility of Renal Safety Biomarkers during Iron Chelation Therapy in Patients with β-Thalassemia and Other Anemias

Explore: A Prospective, Multinational Natural History Study of Patients with Acute Hepatic Porphyrias (AHP) with Recurrent Attacks

Gene Therapy for Beta Thalassemia: Preliminary Results from the PHASE I/II Tiget-Bthal Trial of Autologous Hematopoietic Stem Cells Genetically Modified with GLOBE Lentiviral Vector

Digital PCR Assay to Assess the Alternative Splicing and Absolute Ferrochelatase Expression

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American Society of Hematology

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American Society of Hematology

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